Ignite Creation Date:
2025-12-25 @ 4:40 AM
Ignite Modification Date:
2025-12-26 @ 3:42 AM
Study NCT ID:
NCT04055818
Status:
RECRUITING
Last Update Posted:
2025-05-25
First Post:
2019-08-12
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
A Study of Nicotinamide With Oral Tetrahydrouridine and Decitabine to Treat High Risk Sickle Cell Disease
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D000755', 'term': 'Anemia, Sickle Cell'}], 'ancestors': [{'id': 'D000745', 'term': 'Anemia, Hemolytic, Congenital'}, {'id': 'D000743', 'term': 'Anemia, Hemolytic'}, {'id': 'D000740', 'term': 'Anemia'}, {'id': 'D006402', 'term': 'Hematologic Diseases'}, {'id': 'D006425', 'term': 'Hemic and Lymphatic Diseases'}, {'id': 'D006453', 'term': 'Hemoglobinopathies'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D009536', 'term': 'Niacinamide'}, {'id': 'D000077209', 'term': 'Decitabine'}, {'id': 'D013767', 'term': 'Tetrahydrouridine'}], 'ancestors': [{'id': 'D009539', 'term': 'Nicotinic Acids'}, {'id': 'D000147', 'term': 'Acids, Heterocyclic'}, {'id': 'D006571', 'term': 'Heterocyclic Compounds'}, {'id': 'D011725', 'term': 'Pyridines'}, {'id': 'D006573', 'term': 'Heterocyclic Compounds, 1-Ring'}, {'id': 'D001374', 'term': 'Azacitidine'}, {'id': 'D001372', 'term': 'Aza Compounds'}, {'id': 'D009930', 'term': 'Organic Chemicals'}, {'id': 'D003562', 'term': 'Cytidine'}, {'id': 'D011741', 'term': 'Pyrimidine Nucleosides'}, {'id': 'D011743', 'term': 'Pyrimidines'}, {'id': 'D009705', 'term': 'Nucleosides'}, {'id': 'D009706', 'term': 'Nucleic Acids, Nucleotides, and Nucleosides'}, {'id': 'D012263', 'term': 'Ribonucleosides'}, {'id': 'D014529', 'term': 'Uridine'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL', 'interventionModelDescription': '1:1 Randomization'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 20}}, 'statusModule': {'overallStatus': 'RECRUITING', 'startDateStruct': {'date': '2020-01-24', 'type': 'ACTUAL'}, 'statusVerifiedDate': '2025-05', 'completionDateStruct': {'date': '2027-04-30', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-05-21', 'studyFirstSubmitDate': '2019-08-12', 'studyFirstSubmitQcDate': '2019-08-12', 'lastUpdatePostDateStruct': {'date': '2025-05-25', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2019-08-14', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2026-10-30', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Blood Hemoglobin', 'timeFrame': '12 weeks', 'description': 'Measure hemoglobin function'}]}, 'oversightModule': {'oversightHasDmc': False, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Sickle Cell Disease']}, 'descriptionModule': {'briefSummary': 'A randomized control trial in 20 subjects with sickle cell disease comparing oral THU-decitabine to nicotinamide and in combination (THU, decitabine and nicotinamide).', 'detailedDescription': 'A randomized control trial comparing oral THU-decitabine to nicotinamide (1:1 randomization), and then comparing the effects of the combination of nicotinamide with THU-decitabine vs either treatment alone. Treatment with each agent alone is for 12 weeks followed by the combination for a further 12 weeks. Patients have the option to enter an extension phase of combination treatment for an additional 24 weeks (total of 48 weeks)'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Age 18 years or older.\n* Written informed consent provided by the subject before study entry.\n* Confirmed sickle cell disease (SCD) as determined by hemoglobin electrophoresis or liquid chromatography.\n* Subject is in his/her steady state and not having any acute complication due to SCD (i.e., hospitalization, acute pain, or acute chest syndrome in the past 14 days).\n* Weight at least 40kg\n* Regular compliance with comprehensive care and previous therapy.\n* Symptomatic SCD is defined as having one of following, despite at least 6 months of hydroxyurea therapy, or refuse to take hydroxyurea for personal reasons: fetal hemoglobin \\<0.5 g/dL, or 3 or more pain episodes per year requiring parenteral narcotics, or 1 or more acute chest syndrome episodes, or Hemoglobin \\<9 g/dL and absolute reticulocyte count \\<250,000/mm3.\n\nExclusion Criteria:\n\n* Inability to give informed consent.\n* Experienced severe sepsis or septic shock within the previous 12 weeks.\n* Last HU dose was ingested within the previous 4 weeks.\n* Currently pregnant or breast-feeding.\n* Alanine Aminotransferase (ALT) ≥ 3 times the upper limit of normal or albumin \\<2.0 mg/dL or direct (conjugated) bilirubin ≥ 1.5 mg/dl.\n* Serum creatinine \\>2.9 mg/dL and calculated creatinine clearance \\<30 mL/min.\n* Platelet count \\>800 x 109/L.\n* Absolute neutrophil count \\<1.5 x 109/L.\n* Female of active childbearing potential who is unwilling to use at least one of the two following forms of birth control: (i) not having heterosexual sexual contact beginning at the screening visit and continuing until 4 weeks after the last dose of decitabine OR (ii) intrauterine device (IUD).\n* Sexually active male who is unwilling to use a condom when engaging in any sexual contact with a female with child-bearing potential, beginning at the screening visit and continuing until 4 weeks after taking the last dose of THU and decitabine. This requirement applies also to males who have had a successful vasectomy.\n* Altered mental status or recurrent seizures requiring anti-seizure medications.\n* Moribund or any concurrent disease (e.g., hepatic, renal, cardiac, metabolic) of such severity that death within 24 weeks is likely.\n* Concurrent diagnosis of malignancy including known Myelodysplastic syndrome, leukemia, or an abnormal karyotype.\n* New York Heart Association (NYHA) class III/IV status.\n* Eastern Co-operative Oncology Group (ECOG) performance status ≥3.\n* Participant is on chronic transfusion therapy\n* Known history of illicit drug or alcohol abuse within the past 12 months.\n* Other experimental or investigational drug therapy in the past 28 days.\n* Taking l-glutamine within the last 28 days\n* Being positive for HIV infection'}, 'identificationModule': {'nctId': 'NCT04055818', 'briefTitle': 'A Study of Nicotinamide With Oral Tetrahydrouridine and Decitabine to Treat High Risk Sickle Cell Disease', 'organization': {'class': 'INDUSTRY', 'fullName': 'EpiDestiny, Inc.'}, 'officialTitle': 'Proof-of-concept Study of Nicotinamide and Oral Tetrahydrouridine (THU) and Decitabine to Treat High Risk Sickle Cell Disease', 'orgStudyIdInfo': {'id': '2019-0631'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Nicotinamide', 'description': 'Oral Nicotinamide 1000 mg twice daily', 'interventionNames': ['Drug: Nicotinamide']}, {'type': 'EXPERIMENTAL', 'label': 'THU Decitabine', 'description': 'Oral 250 mg THU and 5 mg decitabine Once per week', 'interventionNames': ['Drug: Nicotinamide']}], 'interventions': [{'name': 'Nicotinamide', 'type': 'DRUG', 'otherNames': ['Decitabine', 'Tetrahydrouridine'], 'description': 'Oral nicotinamide (Vitamin B3) alone compared to THU Decitabine combination', 'armGroupLabels': ['Nicotinamide', 'THU Decitabine']}]}, 'contactsLocationsModule': {'locations': [{'zip': '60612', 'city': 'Chicago', 'state': 'Illinois', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Lani Krauz', 'role': 'CONTACT', 'email': 'Llgnacio@UIC.EDU', 'phone': '312-413-0242'}], 'facility': 'University of Illinois at Chicago College of Medicine', 'geoPoint': {'lat': 41.85003, 'lon': -87.65005}}], 'centralContacts': [{'name': 'Lani Krauz', 'role': 'CONTACT', 'email': 'LIgnacio@UIC.EDU', 'phone': '312-413-0242'}], 'overallOfficials': [{'name': 'Robert Molokie', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'University of Illinois at Chicago College of Medicine'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'EpiDestiny, Inc.', 'class': 'INDUSTRY'}, 'collaborators': [{'name': 'National Institutes of Health (NIH)', 'class': 'NIH'}, {'name': 'National Heart, Lung, and Blood Institute (NHLBI)', 'class': 'NIH'}], 'responsibleParty': {'type': 'SPONSOR'}}}}